Faron Pharmaceuticals has announced initial positive data from the Phase II segment of the ongoing BEXMAB clinical trial of bexmarilimab in combination with azacytidine in relapsed/refractory myelodysplastic syndrome (r/r MDS).

The trial enrolled MDS patients who had failed treatment with hypomethylating agents (HMA).

This trial phase follows a high overall response rate (ORR) of 87.5% from the Phase I portion.

In the combined Phase I and II portions of the BEXMAB trial, a total of 14 HMA-failed MDS patients have been treated with bexmarilimab plus azacitidine.

According to the latest findings, the combination treatment was found to be well tolerated without demonstrating any dose-limiting toxicity.

The ORR for this patient group is reported at 79% with a true remission rate of 64%.

The reported outcomes for the 14 patients include a complete response (CR), seven marrow complete remissions (mCR), one partial response (PR), two haematological improvements, two stable diseases (SD), and one progressive disease (PD).

These results are notably higher than the 0%-20% ORR typically reported with currently available treatment alternatives.

Two of the trial subjects have progressed to bone marrow transplantation, which is considered a potential curative treatment. This progression to transplantation is relatively rare for this patient demographic, as achieving remission is typically challenging.

For patients from the Phase I portion with adequate follow-up, the estimated median overall survival (mOS) is currently at 13.4 months, although this figure is subject to change.

Faron Pharmaceuticals CEO Dr Juho Jalkanen said: “This is a significant milestone for Faron. Many of us have recognised the grave need for new treatment options in r/r MDS.

“With these first results from Phase II continuing the positive results already seen in Phase I, we are committed to rapidly advancing Bexmarilimab to market, because patients are waiting for treatment options like this.”